M-STAR: Study of BHV-3241 in Subjects With Multiple System Atrophy

Sponsor

Biohaven Pharmaceuticals, Inc. (Industry)

Overall Status

Completed

CT.gov ID

NCT03952806

Collaborator

(none)

336

Enrollment

Locations

Arms

35.1

Actual Duration (Months)

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of this study is to compare the efficacy of BHV-3241 versus placebo in subjects with Multiple System Atrophy

Condition or Disease	Intervention/Treatment	Phase
Multiple System Atrophy	Drug: Verdiperstat Drug: Placebo	Phase 3

Study Design

Study Type:

Interventional

Actual Enrollment :

336 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Triple (Participant, Care Provider, Investigator)

Masking Description:

Double-blind to Sponsor, Investigator and Subject

Primary Purpose:

Treatment

Official Title:

A Randomized, Double-Blind, Placebo-Controlled, Parallel- Group Study to Evaluate the Efficacy and Safety of BHV-3241 in Subjects With Multiple System Atrophy (M-STAR Study)

Actual Study Start Date :

Jul 29, 2019

Actual Primary Completion Date :

Jul 29, 2021

Actual Study Completion Date :

Jun 30, 2022

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Arm 1: BHV-3241- Experimental	Drug: Verdiperstat 300mg 2 -oral- capsules, BID
Placebo Comparator: Arm 2: Placebo Comparator	Drug: Placebo Matching placebo

Arm

Intervention/Treatment

Experimental: Arm 1: BHV-3241- Experimental

Drug: Verdiperstat

300mg 2 -oral- capsules, BID

Placebo Comparator: Arm 2: Placebo Comparator

Drug: Placebo

Matching placebo

Outcome Measures

Primary Outcome Measures

Evaluate the efficacy of BHV-3241, compared to placebo, as measured by a change from baseline in a modified Unified MSA Rating Scale (UMSARS) score at Week 48. [Change from Baseline to Week 48]
Measured by a change from baseline in a modified Unified MSA Rating Scale (UMSARS) score.
Assess the safety and tolerability of BHV-3241, relative to placebo, in subjects with MSA. [Change from Baseline to Week 48]
Measured by the difference in amount and severity of adverse events including clinically significant abnormal laboratory values, and electrocardiogram (ECG) results.

Secondary Outcome Measures

Evaluate the efficacy of BHV-3241, compared to placebo, as measured by the Clinical Global Impression of Improvement (CGI-I) score at Week 48. [Change from Baseline Week 48]
Measured by the Clinical Global Impression of Improvement (CGI-I) score
Evaluate the impact of BHV-3241 on quality of life, compared to placebo, as measured by a change from baseline in the motor and non-motor subscales of the MSA- QoL scale at Week 48. [Change from Baseline to Week 48]
Measured by a change from baseline in the motor and non-motor subscales of the MSA- QoL scale.
Evaluate the efficacy of BHV-3241, compared to placebo, as measured by a change from baseline in the UMSARS Part I and Part II total score at Week 48. [Change from Baseline to Week 48]
Measured by a change from baseline in the UMSARS Part I and Part II total score

Eligibility Criteria

Criteria

Ages Eligible for Study:

40 Years to 80 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Diagnosis of probable or possible MSA according to consensus clinical criteria (Gilman et al 2008), including subjects with MSA of either subtype (MSA-P or MSA-C).
Able to ambulate without the assistance of another person, defined as the ability to take at least 10 steps. Use of assistive devices (e.g., walker or cane) is allowed.
Anticipated survival of at least 3 years at the time of Screening, as judged by the Investigator.

Exclusion Criteria:

Any condition that would interfere with the subject's ability to comply with study instructions, place the subject at unacceptable risk, and/or confound the interpretation of safety or efficacy data from the study, as judged by the Investigator.
Diagnosis of neurological disorders, other than MSA.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Barrow Neurological Institute	Phoenix	Arizona	United States	80013
2	UC San Diego Department of Neurosciences	La Jolla	California	United States	92037
3	UCLA Medical Center / Neurological Services	Los Angeles	California	United States	90095
4	Stanford University	Palo Alto	California	United States	84127
5	UCSF Memory and Aging Center	San Francisco	California	United States	95158
6	Rocky Mountain Movement Disorders Center	Englewood	Colorado	United States	80113
7	Parkinson's Disease and Movement Disorders Center of Boca Raton	Boca Raton	Florida	United States	33486
8	University of Florida	Gainesville	Florida	United States	32611
9	Mayo Clinic Florida	Jacksonville	Florida	United States	32224
10	University of South Florida	Tampa	Florida	United States	33612
11	Emory University	Atlanta	Georgia	United States	30329
12	Rush University Medical Center	Chicago	Illinois	United States	60612
13	University of Chicago	Chicago	Illinois	United States	60637
14	University of Kansas Medical Center	Kansas City	Kansas	United States	66160
15	John Hopkins University	Baltimore	Maryland	United States	21287
16	Massachusetts General Hospital	Boston	Massachusetts	United States	02114
17	Beth Israel Deaconess Medical Center	Boston	Massachusetts	United States	02215
18	Lahey Hospital & Medical Center	Burlington	Massachusetts	United States	01805
19	QUEST Research Institute	Farmington Hills	Michigan	United States	48334
20	Mayo Clinic	Rochester	Minnesota	United States	55905
21	Albany Medical College	Albany	New York	United States	12208
22	NYU School of Medicine, NYU Dysautonomia Center	New York	New York	United States	10016
23	Columbia University Medical Center, Neurological Institute	New York	New York	United States	10032
24	Pennsylvania State University Hershey Medical Center	Hershey	Pennsylvania	United States	17033
25	Parkinson's Disease and Movement Disorders Center at the University of Pennsylvania	Philadelphia	Pennsylvania	United States	19107
26	Vanderbilt University Medical Center	Nashville	Tennessee	United States	37232
27	Kerwin Research Center	Dallas	Texas	United States	75231
28	UT Southwestern Medical Center	Dallas	Texas	United States	75390
29	Swedish Medical Center	Seattle	Washington	United States	98122
30	Confraternitaet Privatklinik Josefstadt in Wien	Wien	Vienna	Austria	1080
31	University Clinic Innsbruck	Innsbruck		Austria	06020
32	CHU de Bordeaux, Service de Neurologie	Bordeaux		France	33076
33	CHU - Hospital de la Timone	Marseille Cedex 5		France	13385
34	Unité d'investigation clinique de Neurologie Rez-de-jardin, Bloc Hopital CHU Pontchaillou	Rennes		France	35033
35	Hopitaux Universitaire de Strasbourg-Centre de References des Maladies Autoimmunes	Strasbourg		France	67098
36	CHU Purpan	Toulouse cedex 9		France	31059
37	University Hospital of Liepzig	Leipzig	Sachsen	Germany	04103
38	St. Josef - Hospital Bochum, Kardiologische Studienambulanz	Bochum		Germany	44791
39	Deutsches Zentrum für Neurodegenerative Erkrankungen (DZNE)	Bonn		Germany	53127
40	University Hospital Duesseldorf	Dusseldorf		Germany	40225
41	CRC Core Facility Medizinische Hochschule Hannover (MHH)	Hannover		Germany	30625
42	Paracelsus-Elena-Klinik	Kassel		Germany	34128
43	Klinik für Neurologie - UKSH - Campus Kiel	Kiel		Germany	24105
44	Universitaetsklinikum Giessen und Marburg GmbH - Parkinson-Studienzentrum, Klinik für Neurologie	Marburg		Germany	35043
45	Universitaetsklinikum Muenster	Muenster		Germany	48149
46	Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano	Milano	Milan	Italy	20122
47	A.O.U. San Giovanni di Dio e Ruggi d'Aragona	Salerno		Italy	84100
48	The Newcastle upon Tyne Hospitals NHS Foundation Trust - Campus for Ageing and Vitality (NGH)	Newcastle Upon Tyne		United Kingdom	NE4 5PL

Sponsors and Collaborators

Biohaven Pharmaceuticals, Inc.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Biohaven Pharmaceuticals, Inc.

ClinicalTrials.gov Identifier:

NCT03952806

Other Study ID Numbers:

BHV3241-301

First Posted:

May 16, 2019

Last Update Posted:

Jul 28, 2022

Last Verified:

Jul 1, 2022

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Keywords provided by Biohaven Pharmaceuticals, Inc.

Multiple System Atrophy (MSA)

Additional relevant MeSH terms:

Multiple System Atrophy

Shy-Drager Syndrome

Atrophy

Study Results

No Results Posted as of Jul 28, 2022